Gene Therapy Boosts Motor Skills, Life Quality in Young Patients

Gene Therapy Boosts Motor Skills, Life Quality in Young Patients

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Gene Therapy Boosts Motor Skills, Life Quality in Young Patients

A gene therapy dramatically improved the motor functions and quality of life in children with aromatic L-amino acid decarboxylase (AADC) deficiency in a recent Phase 1 clinical trial. The healthy copy of the AADC gene that the children received corrected the seizure-like episodes associated with the disorder and, in many cases, enabled the children to develop normal head control, sit independently, and walk with support. The study, “Gene therapy for aromatic L-amino acid decarboxylase deficiency…

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